On June 11, 2025 Kazia Therapeutics Limited (NASDAQ: KZIA), a clinical-stage biotechnology company pioneering next-generation oncology therapeutics, reported the publication of transformative preclinical research in the journal Molecular Cancer Therapeutics, a journal of the American Association for Cancer Research (AACR) (Free AACR Whitepaper) that underscores the powerful potential of its lead asset, paxalisib, in reshaping the treatment landscape for triple-negative breast cancer (TNBC), one of the most aggressive and treatment-resistant cancer subtypes (Press release, Kazia Therapeutics, JUN 11, 2025, View Source [SID1234653823]).
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The preclinical program was conducted by Professor Sudha Rao at the renowned QIMR Berghofer Medical Research Institute, revealing how paxalisib can reprogram the tumor microenvironment and enhance immune response, showing substantial synergy with immune checkpoint inhibitors. The data provide strong scientific and translational rationale for continued development of paxalisib as part of immunotherapy-based regimens.
Key Findings
Dual targeting of PI3K and mTOR but not PI3K alone inhibits cancer cell proliferation and migration in vitro.
Paxalisib remodels the TNBC tumor microenvironment, increasing CD4+ and CD8+ T cell infiltration and activation
The combination of paxalisib with KEYTRUDA (pembrolizumab) demonstrated synergistic antitumor activity in advanced breast cancer, resulting in robust tumor regression and prolonged survival in preclinical models
The data validate a mechanistic rationale for ongoing clinical exploration of paxalisib in combination with checkpoint inhibitors and PARP inhibitors
"This landmark study offers a mechanistic and translational foundation for our newly launched Phase 1b clinical trial of paxalisib in advanced breast cancer," said Dr. John Friend, CEO of Kazia. "It not only extends the therapeutic potential of paxalisib beyond brain cancers but also positions it at the forefront of innovative immunotherapy combinations in solid tumors."
The publication, "Combination of the PI3K/mTOR inhibitor paxalisib with immune checkpoint inhibitors enhances antitumor activity in preclinical models of triple-negative breast cancer," is available online at View Source
Clinical Milestone
Kazia recently announced that the first patient has been dosed in the Phase 1b trial evaluating paxalisib in combination with checkpoint inhibitors and chemotherapy in advanced breast cancer, marking a critical step toward clinical translation of these findings.
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